Effect of anticholinergics vs. alpha-1 blockers vs. combination therapy on bladder function in patients with posterior urethral valve after transurethral fulguration: a pilot randomized controlled trial

Clinical Snapshot

Key Findings

• P Value: No statistically significant between-group differences in baseline-adjusted exploratory analyses

• Effect Size: Group I: significant improvement in voided volume; Group III showed most consistent favourable pattern for cystometric capacity and maximum detrusor pressure

• Primary Outcome: Urodynamic parameters showed favourable within-group trends across all treatment arms, with combination therapy demonstrating most consistent pattern

• Nnt Or Sensitivity: Not calculable from pilot study data - hypothesis-generating results only

• Confidence Interval: Wide confidence intervals reported across groups, most overlapping null value

Clinical Application

High feasibility demonstrated with 81% recruitment rate and 91% urodynamic study completion in specialised centres Relevant to Australian paediatric urology practice. Anticholinergics and alpha-1 blockers available on PBS for appropriate indications. Would require specialist paediatric urology assessment and urodynamic facilities available in major Australian children's hospitals Boys under 12 years with bladder dysfunction following PUV ablation in tertiary paediatric centres

Abstract

PURPOSE: To identify preliminary efficacy signals of anticholinergics, alpha-1 blockers, and their combination on bladder and renal function in boys with bladder dysfunction following posterior urethral valve (PUV) ablation, and to assess the feasibility of conducting a definitive randomized, controlled trial. STUDY DESIGN: In this single-center, prospective, randomized pilot trial boys under 12 years who had undergone cystoscopic valve ablation were randomized into three groups: anticholinergic therapy (Group I), alpha-1 blocker therapy (Group II), and combination therapy (Group III). The study was designed to detect within-group preliminary efficacy signals in urodynamic parameters following 6 months of pharmacotherapy. Secondary outcomes included changes in patient-reported symptom scores, renal function, and feasibility metrics. Between-group comparisons were performed as exploratory analyses. Feasibility and safety outcomes were analyzed in the intention-to-treat population, while efficacy analyses were conducted in participants who received intervention and had evaluable baseline and follow-up data. RESULTS: Thirty-five eligible patients were randomized (Group I, n = 12; Group II, n = 11; Group III, n = 12). and their clinical and biochemical analyses at baseline were recorded. Two deaths in Group II occurred prior to baseline urodynamics and pharmacotherapy initiation; efficacy analyses, therefore, included 33 patients (Group I, n = 12; Group II, n = 9; Group III, n = 12) with evaluable data. Urodynamic evaluation demonstrated favourable within-group trends: Group I showed a significant improvement in voided volume, while changes in voiding time, cystometric capacity (CC), static compliance, and maximum detrusor pressure (Pdetmax) were favourable but not uniformly statistically significant. Group II showed modest, favourable effects on Qmax, Time@Qmax, CC, and Pdetmax, with wide confidence intervals and no clear significant changes across most UDS measures. Group III demonstrated the most consistent favourable pattern, particularly for CC and Pdetmax, although most confidence intervals overlapped the null. Baseline-adjusted exploratory analyses did not demonstrate statistically significant between-group differences. Symptom scores improved across all groups. Feasibility outcomes were robust, with 81% recruitment, 91% urodynamic study completion, and 94% participant retention. CONCLUSION: This pilot trial demonstrates the feasibility of conducting urodynamic-guided pharmacotherapy studies in young boys with PUV-related bladder dysfunction. The observed within-group efficacy signals across all treatment arms-particularly with combination therapy-are hypothesis-generating and provide effect size and variability estimates to inform the design of a definitive randomized controlled trial.

References

1. 1.Singhai, P., Malik, M. A., & Mahajan, J. K. (2024). Effect of anticholinergics vs. alpha-1 blockers vs. combination therapy on bladder function in patients with posterior urethral valve after transurethral fulguration: a pilot randomized controlled trial. Journal of Pediatric Urology. https://doi.org/10.1016/j.jpurol.2024.06.005